Opal Sandy Receives Groundbreaking Gene Therapy Treatment, Hears for the First Time
Opal Sandy, a deaf child from Oxfordshire, UK, has become the first British patient and youngest child in the world to undergo groundbreaking gene therapy treatment that has allowed her to hear for the first time. Opal was born deaf due to a rare genetic disorder called auditory neuropathy, which affects the inner ear’s ability to communicate with the hearing nerve.
The treatment involved an infusion of a harmless virus containing a working copy of the OTOF gene into Opal’s cochlea, along with a cochlear implant in her right ear. According to her mother, Jo Sandy, the results have been “mind-blowing” as Opal can now hear soft sounds and whispers in her treated ear, and is responding to her parent’s voices and communicating words.
The trial, which began in May 2023, is expected to include up to 18 children and could have a global impact on the treatment of genetic causes of deafness. Opal’s case will be presented at the annual meeting of the American Society of Gene and Cell Therapy, demonstrating the potential of gene therapy in cases of genetic hearing loss.
This groundbreaking treatment has given hope to many families affected by genetic hearing loss and highlights the importance of continued research and advancements in the field of gene therapy. Opal’s story serves as a beacon of hope for those who are facing similar challenges and paves the way for a brighter future for those living with genetic disorders causing deafness.
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